May 27, 2015
TREAT-NMD Advisory Committee for Therapeutics (TACT) was established to provide independent and objective guidance on the preclinical and development pathway of potential therapies (whether novel or repurposed) for neuromuscular diseases. A review published in Orphanet Journal of Rare Diseases presents the TACT’s experience of providing experts from multiple disciplines to help sponsors test their candidate compounds according to a realistic and well-informed clinical trial plan, and eventually obtain registration.
The article details the stringent membership procedures for TACT, currently composed of 67 international experts in various areas of drug development, representing 11 countries. The article states that TACT has reviewed 29 programme applications in several rare neuromuscular diseases from industry and academia, including novel and repurposed medications in preclinical and clinical stage applications. Out of these applications, TACT has helped gain orphan drug designation from the EMA and FDA for three products each. The authors also describe the recurrent themes of these applications over the past five years and believe their experience can be extrapolated to bring medications for other groups of rare diseases to the market.